Webinar Overview

The past decade has seen numerous innovations in novel therapies for rare diseases, providing clinical and humanistic benefits for populations that are small but have large unmet need. Payers and manufacturers alike recognize the high price of innovation, and healthcare systems have struggled in recent years to manage the budget impact of life-changing treatments for patients with rare diseases.

This webinar explores these issues, highlighting opportunities, challenges, presenting real world examples and providing practical guidance. We will focus on:

  • Ethics and politics of orphan disease treatment
  • Current global payer perspectives on treatments for rare diseases
  • Strategies and solutions for developing an evidence-based value proposition that will resonate with key stakeholders (payers, clinicians, patient advocates)


Who Should Attend

Managers, Directors and others in the pharmaceutical industry in the areas of:

  • HEOR (health economics/outcomes research)
  • Medical affairs
  • Epidemiology
  • Market access
  • Pricing and reimbursement

Key Learning Objectives

  • Describe the rationale for providing life-changing but costly therapies for patients with rare diseases
  • Recognize the key decision-making criteria for global payers in evaluating therapies for rare diseases
  • Identify key components of an evidence-based value proposition that would support market access for a treatment for a rare disease


Thomas Rising, DPhil, Managing Consultant, Global Payer Research and Strategy, Evidera

Alex Ward, PhD, MRPharmS, Senior Research Scientist, Evidera

Karen Sandman, PhD, US Practice Lead, Global Market Access Communications, Evidera